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[Comment] A hopeful therapy for Niemann-Pick C diseases

21:22 EDT 10 Aug 2017 | The Lancet

Niemann-Pick C1 disease (NPC1) is a rare autosomal recessive lysosomal storage disease, which was separated from the sphinomyelinase-deficient NPCA and NPCB when cholesterol was found to be stored.1 No drugs for the disease are currently approved in the USA, although miglustat is approved in Europe. In The Lancet, Daniel Ory and colleagues2 report strong evidence that intrathecal delivery of hydroxypropyl-beta-cyclodextrin (HPBCD) slows the progression of NPC1.

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